ProQR Therapeutics NV

LUXTURNA has significant market potential due to the high unmet need for effective treatments in rare genetic conditions. With its FDA approval for specific mutations in the RPE65 gene, it offers hope to patients who previously had limited options. The growing awareness and adoption of gene therapies in ophthalmology, along with advancements in personalized medicine, are expected to expand LUXTURNA's market reach. LAS VEGAS, March 26, 2025 /PRNewswire/ -- DelveInsight's " LUXTURNA Market Size, Forecast, and Market Insight Report" highlights the details around LUXTURNA, a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of LUXTURNA. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Spark Therapeutics/Novartis' LUXTURNA (AAV2-hRPE65v2; voretigene neparvovec) Overview LUXTURNA (voretigene neparvovec-rzyl, AAV2-hRPE65v2) is a one-time gene therapy designed to treat vision loss caused by mutations in both copies of the RPE65 gene. It works by delivering a functional copy of the RPE65 gene to replace the faulty one, helping to restore vision. The therapy is administered as a single subretinal injection beneath the retina in patients with confirmed RPE65 mutations and viable retinal cells. LUXTURNA was developed and commercialized in the US by Spark Therapeutics. Outside the US, Novartis holds the rights for development, registration, and commercialization under a licensing agreement. It is approved for treating vision loss caused by Leber's congenital amaurosis or retinitis pigmentosa due to confirmed biallelic RPE65 mutations. LUXTURNA uses an adeno-associated viral type 2 (AAV2) vector to deliver the RPE65 gene. The vector includes a cytomegalovirus (CMV) enhancer and a chicken beta-actin promoter to drive the expression of the normal human RPE65 protein. The parent AAV2 virus, which serves as the template, is a non-pathogenic, single-stranded DNA virus that depends on a helper virus for replication. Once inside retinal cells, the functional RPE65 protein enables the regeneration of 11-cis-retinal, which improves light detection and restores vision. In healthy individuals, RPE65 in retinal pigment epithelial cells converts all-trans-retinol into 11-cis-retinol, which then forms 11-cis-retinal—a critical step in the visual cycle that converts light into electrical signals. Mutations in the RPE65 gene disrupt this process, leading to vision loss. LUXTURNA restores the retinoid cycle by reintroducing functional RPE65 protein. LUXTURNA Dosage and Administration The recommended dose for each eye is 1.5 × 10¹¹ vector genomes (vg) in a 0.3 mL volume, administered via subretinal injection. Each eye should be treated on different days, with at least six days between injections. Oral corticosteroids equivalent to prednisone at 1 mg/kg/day (up to 40 mg/day) should be given for seven days, starting three days before injection, followed by a tapering dose over the next 10 days. LUXTURNA Dosage Forms and Strengths LUXTURNA is provided as a suspension for subretinal injection in a 0.5 mL extractable volume within a 2 mL single-dose vial. The concentration is 5 × 10¹² vg/mL, requiring a 1:10 dilution before use. The diluent is supplied in two single-use 2 mL vials. Learn more about LUXTURNA projected market size for retinitis pigmentosa @ LUXTURNA Market Potential Retinitis pigmentosa is a group of inherited retinal disorders that cause progressive degeneration of the retina and eventual blindness. It involves the gradual bilateral deterioration of rod and cone photoreceptors, leading to night blindness and progressive loss of peripheral vision, followed by a decline in central vision due to the loss of cone function. Retinitis pigmentosa is primarily linked to genetic mutations, with over 44 associated genes identified. In 2023, approximately 113,000 people in the US were affected by retinitis pigmentosa. Currently, available treatments are mainly off-label and do not address the underlying genetic causes of retinitis pigmentosa. Patients without the RPE65 mutation rely on supportive care, such as vitamin supplements, protection from sunlight, and visual aids. LUXTURNA is the only approved therapy for retinitis pigmentosa but is limited to patients with the RPE65 mutation. Gene and cell therapies are becoming key treatment strategies, targeting genes like RHO, USH2A, and RPGR. USH2A is a major focus, with two oligonucleotide candidates advancing in the RP pipeline. Optogenetics offers a novel gene therapy approach that works independently of specific gene mutations and shows promise in treating late-stage RP with severe photoreceptor loss. Several companies have progressed optogenetic gene therapies into clinical trials. According to DelveInsight, the retinitis pigmentosa gene therapy market is expected to grow due to increasing disease prevalence, improved awareness and treatment access, and a strong pipeline of therapies across the 7MM. Discover more about the retinitis pigmentosa market in detail @ Retinitis Pigmentosa Market Report Emerging Competitors of LUXTURNA Some of the drugs in the retinitis pigmentosa pipeline include Botaretigene sparoparvovec (Johnson & Johnson Innovative Medicine/MeiraGT), AGTC-501 (Beacon Therapeutics), MCO-010 (Nanoscope Therapeutics), GS030 (Gensight Biologics), CTx PDE6B (Coave Therapeutics), OCU 400 (Ocugen), jCell (jCyte), EA-2353 (Endogena Therapeutics), Ultevursen (ProQR Therapeutics), ADX‑2191 (Aldeyra Therapeutics), and other. In January 2025, Beacon Therapeutics Holdings Limited reported that the FDA has awarded Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (laruparetigene zovaparvovec) for treating X-linked retinitis pigmentosa (XLRP). To know more about the number of competing drugs in development, visit @ LUXTURNA Market Positioning Compared to Other Drugs Key Milestones of LUXTURNA In October 2023, SpliceBio entered an exclusive collaboration and licensing agreement with Spark Therapeutics to utilize SpliceBio's proprietary Protein Splicing platform to develop a gene therapy for an undisclosed inherited retinal disease. In June 2023, Novartis Pharma obtained manufacturing and marketing approval for LUXTURNA in Japan as a gene therapy for IRD caused by mutations in both copies of the RPE65 gene. In November 2018, Novartis announced that the European Commission (EU) approved LUXTURNA for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene and who have enough viable retinal cells. The authorization is valid in all 28 member states of the EU, as well as Iceland, Liechtenstein, and Norway. In January 2018, Novartis entered into a licensing agreement with Spark Therapeutics covering development, registration, and commercialization rights to voretigene neparvovec in markets outside the US. Spark Therapeutics retains US rights for LUXTURNA. In December 2017, the FDA approved Spark Therapeutics' LUXTURNA for patients with confirmed Bi-allelic RPE65 Mutation-associated Retinal Dystrophy. In July 2017, Spark Therapeutics announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the US FDA designated LUXTURNA as a drug for a rare pediatric disease. In October 2016, LUXTURNA granted ODD for the treatment of inherited retinal dystrophy due to bi-allelic RPE65 mutation. In July 2015, LUXTURNA was designated as an orphan medicinal product for the treatment of retinitis pigmentosa. In September 2014, the US FDA granted LUXTURNA a Breakthrough Therapy Designation for Inherited retinal dystrophy. LUXTURNA Patent Details The company holds the following patents related to its AAV-based retinal gene therapy: US patent application relating to cell transfection improvements for AAV vector production. We expect that any patents issued from this application will expire in 2038, excluding any potential patent term extension or adjustment. A US patent application relating to improvements in AAV vector purification. It expects that any patent from this application will expire in 2038, excluding any potential patent term extension or adjustment. Patent for manufacturing gene therapy vector Spark Therapeutics holds exclusive licenses for three patent application families from CHOP, covering scalable methods for manufacturing high-purity gene therapy vectors. The first patent family pertains to the production of Spark's product candidates, including those under its collaboration with Pfizer. Patents from this family have been granted in the United States, Australia, and Mexico, with expiration dates in 2021, not accounting for any potential extensions or adjustments. Related patent applications are still under review in the United States, Brazil, Canada, China, Europe, Israel, India, and Japan. If granted, these patents are expected to expire in 2031, excluding any possible extensions or adjustments. Modified AAV vectors and gene delivery Spark Therapeutics is advancing technology across multiple areas to enhance and broaden its current product pipeline. This technology, exclusively licensed from CHOP, focuses on modifying gene therapy vectors, incorporating companion therapies or diagnostics, and developing new therapeutic genes. The licensed patent portfolio includes: Six US patent applications covering alternative or modified AAV vectors for gene delivery. Any resulting patents are expected to expire between 2028 and 2034, excluding possible patent term extensions or adjustments. Two pending US patent applications related to inhibiting immune responses to AAV vectors and detecting AAV-binding antibodies. Any patents granted from these applications are anticipated to expire between 2032 and 2034, excluding potential patent term extensions or adjustments. Discover how LUXTURNA is shaping the retinitis pigmentosa treatment landscape @ LUXTURNA Gene Therapy LUXTURNA Market Dynamics The market for LUXTURNA is primarily driven by the increasing prevalence of inherited retinal diseases (IRDs) and the growing acceptance of gene therapy as a viable treatment option. The high unmet medical need for effective treatments for rare genetic diseases has created a favorable environment for LUXTURNA. The therapy's ability to deliver long-term benefits from a single administration, reducing the burden of repeated treatments, has also contributed to its market acceptance. Furthermore, the strong backing from Roche, with its extensive commercialization and market access infrastructure, has helped expand LUXTURNA's reach across multiple markets. Favorable reimbursement policies in the U.S. and Europe have further supported market uptake. Despite its success, LUXTURNA faces significant market challenges. The high cost of treatment, which exceeds $850,000 for both eyes, poses a barrier to widespread adoption, even with reimbursement support. Access to treatment is also limited by the need for specialized surgical centers and trained ophthalmologists, which restricts the therapy's availability to select geographic regions. Moreover, as gene therapy technology evolves, the emergence of newer, potentially more effective or cost-efficient treatments could pose competitive pressure. Additionally, the complexity of gene therapy manufacturing and delivery, along with stringent regulatory requirements, adds to the operational challenges faced by Spark Therapeutics and Roche. The future market dynamics of LUXTURNA are expected to be influenced by ongoing advancements in gene therapy, increasing diagnostic capabilities for inherited retinal diseases, and expanding treatment infrastructure. Strategic partnerships and continued investment in patient access programs and healthcare provider training could enhance market penetration. Additionally, the successful clinical outcomes and long-term durability of LUXTURNA will play a crucial role in sustaining patient and physician confidence. As the gene therapy market matures, LUXTURNA's position as a first-mover in the inherited retinal disease space will likely provide it with a competitive edge, although it will need to navigate pricing pressures and emerging competition in the broader ophthalmology and gene therapy markets. Dive deeper to get more insight into LUXTURNA's strengths & weaknesses relative to competitors @ LUXTURNA Market Drug Report Table of Contents Related Reports Retinitis Pigmentosa Market Retinitis Pigmentosa Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key retinitis pigmentosa companies including Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, Gensight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Ocugen, Bionic Sight, jCyte, Endogena Therapeutics, ProQR Therapeutics, Aldeyra Therapeutics, among others. Retinitis Pigmentosa Pipeline Retinitis Pigmentosa Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key retinitis pigmentosa companies, including Acucela Inc, Allergan Plc, Amarantus Bioscience Holdings Inc, Amgen Inc, Applied Genetic Technologies Corp, Asklepios BioPharmaceutical Inc, Astellas Pharma Inc, Caladrius Biosciences Inc, Dompe Farmaceutici SpA, GenSight Biologics SA, Grupo Ferrer Internacional SA, IDPharma Co Ltd, Janssen Pharmaceutical, MeiraGTx, Anabasis Pharma, Allegro Ophthalmics, Nacuity Pharmaceuticals, ReNeuron, jCyte, Editas Medicine, SanBio, NightstarTherapeutics, Biogen, OiDE OptoEye, among others. Cell and Gene Therapies in Rare Disorders Market Cell and Gene Therapies in Rare Disorders Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cell and gene therapies in rare disorders companies including REGENXBIO, Coave Therapeutics, GenSight Biologics, Ultragenyx, Pfizer, Sangamo Therapeutics, Roche, 4D Molecular Therapeutics, Astellas Gene Therapie, Actus Therapeutics, Nanoscope Therapeutics, Ocugen, jCyte, Amicus Therapeutics, Capricor Therapeutics, Nippon Shinyaku, Brainstorm Cell Therapeutics, Editas Medicine, Abeona Therapeutics, Ishin Pharma, among others. Gene Therapy Competitive Landscape Gene Therapy Competitive Landscape – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key gene therapy companies, including Novartis, Johnson & Johnson, Fibrocell Technologies, Pfizer, HELIXMITH Co., Ltd., Sarepta Therapeutics, REGENXBIO, Solid Biosciences Inc., Lexeo Therapeutics, Spark Therapeutics, Xalud Therapeutics, uniQure, Ultragenyx Pharmaceutical, Nanoscope Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company expects up to four clinical data readouts in 2025 and 2026 across multiple programs, highlighted by first clinical data for AX-0810 anticipated in Q4 2025€ 149.4 million cash and cash equivalents as of end of 2024, providing runway into mid-2027, plus additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., March 13, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2024, and provided a business update. “As we continue to advance our Axiomer ADAR-mediated RNA editing platform and pipeline, ProQR is entering a pivotal phase of growth and innovation,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “With up to four clinical trial data readouts anticipated in 2025 and 2026, including topline data for our NTCP program later this year, we believe we are poised to demonstrate the broad therapeutic potential of RNA editing. Our growing discovery pipeline, now including CNS applications, leading intellectual property portfolio, and strategic partnerships – such as our $3.9 billion collaboration with Eli Lilly and partnership with the Rett Syndrome Research Trust – reinforce our leadership in the field.” De Boer continued, “Our successful financing in October 2024 further strengthens our financial foundation, providing cash runway into mid-2027. With this strong position, we believe we are well equipped to advance key upcoming milestones, including submission of the CTA for our first RNA editing pipeline program, AX-0810, on track for Q2 2025 and there following our first clinical data readout in Q4 of this year, as well as continued progress across our pipeline and partnered programs. We remain committed to driving forward the next era of transformative RNA editing therapeutics for patients in need.” Recent Progress In December 2024, the Company hosted a comprehensive virtual Analyst and Investor event to highlight pipeline programs and progress with its proprietary Axiomer RNA editing technology platform.In December 2024, ProQR expanded its collaboration with the Rett Syndrome Research Trust, securing additional funding for a total of up to $9.2 million to support the advancement of AX-2402, an RNA editing therapy targeting the R270X mutation in the MECP2 gene associated with Rett Syndrome, into clinical trials.In December 2024, ProQR appointed Dr. Peter A. Beal, as Chief ADAR Scientist. Dr. Beal is a leading expert in ADAR and RNA biology from the University of California, Davis, and member of ProQR’s Scientific Advisory Board. As Chief ADAR Scientist, he drives the development and optimization of the Company’s Axiomer ADAR-mediated RNA editing platform.In October 2024, ProQR closed an underwritten public offering of 18,000,000 ordinary shares and a concurrent private placement of 3,523,538 ordinary shares with Eli Lilly and Company (Lilly), as well as exercise of the underwriters’ option for 1,940,072 additional shares for gross proceeds totaling approximately $82.1 million. Anticipated Upcoming Events AX-0810 targeting NTCP for Cholestatic diseases Q2 2025 Clinical Trial Application (CTA) submissionQ4 2025 first clinical target engagement and biomarker data in healthy volunteers AX-2402 targeting MECP2 (R270X) for Rett Syndrome 2025 clinical candidate selection 2026 clinical trial initiation and topline data readout AX-2911 targeting PNPLA3 for MASH 2025 clinical candidate selection 2026 clinical trial initiation and topline data readout AX-1412 targeting B4GALT1 for Cardiovascular diseases mid-2025 update on optimization for GalNAc delivery Continue to execute on partnership with Lilly, with potential data updates, milestone income from the existing partnership, and an option to exercise for an additional five targets for expansion to a total of 15 targets, which would result in a $50 million opt-in payment to ProQR.ProQR may selectively form new partnerships, which could include multi-target discovery alliances, or product alliances on specific programs. Year End 2024 Financial Highlights At December 31, 2024, ProQR held cash and cash equivalents of € 149.4 million, compared to € 118.9 million at December 31, 2023. Net cash used in operating activities during the full year ended December 31, 2024 was € 36.4 million, compared to € 21.5 million net cash generated by operating activities for the same period in 2023. The Company experienced a net positive cash flow from operating activities in 2023 primarily due to the receipt of the Lilly up-front payment of $60 million in February 2023. Research and development costs for the year ended December 31, 2024 were € 36.4 million, compared to € 25.1 million for the same period in 2023. The increase in research and development costs for the year ended December 31, 2024 compared to 2023 was due primarily to increased investments in the platform and advancing pipeline programs. General and administrative costs for the year ended December 31, 2024 were € 13.7 million, compared to € 16.2 million for the same period in 2023. Net loss for the year ended December 31, 2024 was € 27.8 million or € 0.32 per diluted share, compared to € 27.7 million, or € 0.35 per diluted share for the year ended December 31, 2023. For further financial information for the period ended December 31, 2024, please refer to our 2024 Annual Report on Form 20-F and our Statutory Annual Report, which will be available on our website, www.proqr.com under Financials and Filings. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com. Forward Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, technology, strategy, preclinical and clinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, the continued advancement of our lead development pipeline programs, including ongoing and planned clinical trials, the anticipated timing of initial clinical data readouts across multiple programs in 2025 and 2026, and the filing of a CTA application for our lead program, AX-0810, in Q2 2025, our Axiomer™ platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates, the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto, our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development, the potential of our technologies and product candidates, the collaboration with Lilly and the intended benefits thereof, including potential milestones, exercise of an option to expand targets and the receipt of an opt-in payment, our ability to selectively form new partnerships and enter into future collaborations, and our financial position and cash-runway. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor and media contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorInvestor contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@lifesciadvisors.com

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 11, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced an expansion of its collaboration with the Rett Syndrome Research Trust (RSRT). Building on the initial $1 million research grant announced in January 2024, the expanded partnership includes an additional $8.1 million in funding from the RSRT, for a total of $9.1 million. The funding will support the advancement of AX-2402 into clinical trials. Rett Syndrome, a rare and devastating neurodevelopmental disorder, affects approximately 350,000 people worldwide, predominantly girls. With no current cures and limited treatment options, Rett Syndrome presents a significant unmet medical need. AX-2402 is being developed for individuals with Rett syndrome who have the R270X mutation in MECP2 gene, and is based on ProQR’s proprietary Axiomer RNA editing platform. Axiomer can target many mutations beyond R270X that collectively impact a large segment of the Rett population. Success with AX-2402 sets the foundation for developing RNA editing therapeutics to target the remaining mutations. “We are deeply committed to bring innovative solutions to patients with high unmet needs, such as those living with Rett Syndrome, and are grateful for the expanded partnership with the Rett Syndrome Research Trust,” said Gerard Platenburg, Chief Scientific Officer of ProQR. “The increased support from RSRT underscores the potential of our Axiomer RNA editing technology platform to address complex genetic CNS conditions. The expanded collaboration enables us to accelerate the development of AX-2402 for Rett syndrome and to advance this program into clinical trials.” Monica Coenraads, Founder and CEO of the Rett Syndrome Research Trust, commented: “Individuals with Rett Syndrome live with the profound effects of this condition every day, and they deserve transformative therapies. This partnership with ProQR is exciting and ambitious. We are encouraged by the progress we’ve seen so far and believe that together we are taking critical steps toward achieving our mission of eradicating Rett Syndrome.” The additional $8.1 million in funding will enable ProQR to accelerate work on RNA editing approaches tailored to the genetic mutations that cause Rett Syndrome. This next phase of the collaboration will focus on optimizing therapeutic candidates targeting the transcription factor Methyl CpG binding protein 2 (MECP2) and advancing them toward clinical development. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. About Rett Syndrome Rett syndrome is a progressive neurodevelopmental disorder caused by genetic mutations in the Methyl CpG binding protein 2 (MECP2) and diagnosed primarily in females. It is characterized by apparently normal psychomotor development during the first six to 18 months after birth, followed by a period of developmental stagnation, then a regression in language and motor skills, followed by long-term relative stability. During the phase of regression, affected patients develop repetitive, stereotypic hand movements that replace purposeful hand use. Additional symptoms include gait ataxia and apraxia, seizures, tremors, episodic apnea and/or hyperpnea, gastrointestinal issues, scoliosis and musculoskeletal problems, anxiety and sleep issues and bruxism. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com. Forward Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, express and implied statements regarding our expanded collaboration with RSRT, including the plan of the collaboration and the intended benefits thereof, the further development and advancement of our Axiomer™ platform, including our anticipated strength and continued investment in it, as well as the potential of our technologies and product candidates, our ability to accelerate the development of AX-2402 for Rett syndrome and advance the program into clinical trials, the therapeutic potential of our Axiomer RNA editing oligonucleotides and our ability to optimize therapeutic candidates covered by the collaboration, and the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto, our business operations, as well as the timing of our clinical development. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Eli Lilly and the Company; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorPeter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@lifesciadvisors.com Media contact:Robert StanislaroFTI ConsultingT: +1 212 850 5657robert.stanislaro@fticonsulting.com